Weekly Quick Hits (Philly) – Week of October 2, 2023

Penn’s Karikó and Weissman win the 2023 Nobel Prize in Medicine, CHOP & Penn researchers receive $50 million NIH grant to study the environmental influence on children’s health and more. Continue reading for more Philadelphia Region life science stories.

By Mark Terry | October 6, 2023

Quick Hits is BioBuzz’s weekly round up of all the life science news you don’t want to miss, in your region of interest. Don’t miss a beat; SUBSCRIBE to our weekly Quick Hits newsletter via LinkedIn (BioHealth Capital Region, Greater Philadelphia, Research Triangle Park) or get it delivered to your inbox every week.

Funding, Awards and Collaborations 

Penn’s Karikó and Weissman Win 2023 Nobel Prize in Medicine

Katalin Karikó, adjunct professor of neurosurgery at the University of Pennsylvania’s Perelman School of Medicine, and Drew Weissman, the Roberts Family Professor of Vaccine Research in the Perelman School of Medicine at the University of Pennsylvania received the 2023 Nobel Prize in Physiology or Medicine. The two Penn researchers’ mRNA research was foundational for the science that underpinned the COVID-19 mRNA vaccines.

Innovation Space and Silicon Catalyst Found Joint Incubator

The Innovation Space (Wilmington, Del.) and Silicon Catalyst (Silicon Valley, Calif.) established a collaboration to co-incubate early-stage science-based enterprises that focus on advanced materials and other enabling technologies needed for the semiconductor industry. Startups can now be considered for lab space at The Innovation Space in Wilmington and/or with virtual support provided by both organizations anywhere in the world.

Immunome and Morphimmune Complete Merger with $125 Million PIPE

Immunome (Seattle) and Morphimmune (Exton, Pa.) announced they had completed their merger. A $125 million private placement investment was completed that the now-merged companies believe will provide a cash runway into the first quarter of 2026. The PIPE included participation from Enavate Sciences, EcoR1 Capital, Redmile Group, Janus Henderson Investors, Avidity Partners, Woodline Partners LP, and other leading institutional investors.

In the Clinic

PDS Biotech’s Phase II Head-and-Neck Cancer Study Shows Promise

PDS Biotechnology Corporation (Princeton, NJ) updated interim data based on an August 2 cutoff from the VERSATILE-002 Phase II trial of PDS0101 in combination with Merck’s Keytruda (pembrolizumab) in patients with unresectable, recurrent, or metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC). The updated data further validates the potential of the combination.

Amicus Presented Updates on Pompe Disease Programs

Amicus Therapeutics (Princeton, NJ) presented four posters describing its development program for Pompe disease at the 28^th Annual Congress of the World Muscle Society (WMS). The company’s Pombiliti (cipaglucosidase alfa) and Opfolda (miglustat) are approved for Pompe disease, a lysosomal acid alpha-glucosidase deficiency.

Trevena Completed Analysis of Olinvyk Continuous Respiratory Monitoring Data from VOLITION Study

Trevena (Chesterbrook, Pa.) announced it had completed the initial analysis of Olinvyk continuous respiratory monitoring data from the VOLITION study. The study is a real-world evaluation of the impact of Olinvyk on respiratory, GI and cognitive function outcomes in the postoperative setting. IV Olinvyk was dosed as the first-line analgesic during postoperative care and administered with a PCA device. Olinvyk is a new chemical entity approved by the FDA in August 2020, which contains the opioid oliceridine.

PDS Biotech’s PDS-0101 + Chemoradiotherapy Linked to Drop in Circulating Tumor DNA

PDS Biotechnology (Princeton, NJ) announced data showing PDS0101 in combination with standard-of-care chemoradiotherapy was linked with a rapid drop in human papillomavirus (HPV) circulating cell-free DNA, a possible predictive biomarker of treatment response. The data was from the IMMUNOCERV Phase II trial. PDS0101 is an HPV-targeted immunotherapy that stimulates a potent targeted T-cell attack against HPV-positive cancers.

SwanBio Moves to Higher-Dose Cohort in Gene Therapy Study for Adrenomyeloneuropathy

SwanBio Therapeutics (Philadelphia) initiated the second dose-escalation cohort of its first interventional clinical Phase I/II PROPEL trial. The trial is evaluating SBT101, an investigational gene therapy designed to compensate for the ABCD1 mutation that causes adrenomyeloneuropathy (AMN). AMN is a progressive and debilitating neurodegenerative disease caused by ABCD1 mutations that disrupt the function of spinal cord cells and other tissues.

FDA Clears Transcenta’s Osemitimab for Phase III Gastric Cancer Study

The FDA gave Transcenta (Princeton, NJ and Suzhou, China) the go-ahead to initiate the TransStar 301 Phase III trial of osemitimab in combination with nivolumab and chemotherapy as first-line treatment in patients with HER2-negative, CLDN18.2 expressing locally advanced or metastatic gastric or gastroesophageal (G/GEJ) adenocarcinoma. Phase III trials were recently approved in South Korea as well.

BriaCell Initiates Phase III Trial of Bria-IMT in Metastatic Breast Cancer

BriaCell Therapeutics (Philadelphia and Vancouver, BC) initiated its Phase III trial of Bria-IMT in combination with an immune checkpoint inhibitor for advanced metastatic breast cancer. Bria-IMT is under FDA Fast Track Designation.

Harmony Biosciences Presents New Prader-Willi Data

Harmony Biosciences (Plymouth Meeting, Pa.) presented new secondary endpoint data from its Phase II signal-detection study of pitolisant for excessive daytime sleepiness in Prader-Willi syndrome. The outcomes data included improvements in behavioral symptoms, especially in the higher-dose group. Decreases in the caregiver rating of EDS severity were also observed.

New Products

Molecular Targeting Technologies Presented EvaThera, Long-Acting Peptide Receptor Radionuclide Therapy Platform

West Chester, Pa.-based Molecular Targeting Technologies presented the company’s EvaThera, the first long-acting, targeted peptide receptor radionuclide therapy (PRRT) platform at BioFuture 2023. PRRT delivers killing radiation directly to cancer cells via unique peptides to target biomarkers in those cancers.

Research Roundup

CHOP & Penn Researchers Receive $50 Million NIH Grant to Study Environmental Influence on Children’s Health

A group of researchers from Children’s Hospital of Philadelphia (CHOP) and Penn Medicine received a $50 million grant from the NIH to study the impact of environmental influences on pregnancy and children’s health. The program is part of NIH’s Environmental Influences on Child Health Outcomes (ECHO) Program.

Penn State Research: New Test for Sepsis for Emergency Departments

A research group at the Penn State College of Health and Human Development found that a new blood test for sepsis could save lives and money. The IntelliSep test identifies changes in a patient’s white blood cells that can determine if the patient is at high risk of sepsis.

Princeton Research: Protein Function Isn’t All About its 3D Shapes

The general idea of protein function is that it’s the stable 3D shapes and how they fit with other biomolecules that are behind their function. However, researchers at the Princeton University Engineering School found that about half of all proteins have “stringy, unstructured” parts that hang off them, called intrinsically disordered regions (IDRs). The research found that IDRs, compared to previous thought, have specific and important interactions that play a key role in chromatin regulation and gene expression.

Menarini Silicon Biosystems Announces New Study of Cellsearch Liquid Biopsy

Menarini Silicon Biosystems (Bologna, Italy and Huntingdon Valley, Pa.) published the results of a clinical trial of the utility of CTCs to detect disease relapse substantially earlier than what is currently possible with routine surveillance imaging, in patients with stage III melanoma. The Menarini Silicon Biosystems CELLSEARCH system and Celltracks Circulating Melanoma Cell Kit were used to analyze the CTCs.

Temple Research: Promising New Therapeutic Target for Alzheimer’s Disease

Researchers at Tempe University’s Alzheimer’s Center published research that identified a promising new therapeutic target, ABCA7, a protein known to protect from Alzheimer’s disease. The research described new data about the relationship between ABCA7, cholesterol, and neuroinflammation.

On the Hill – Regulatory and Advocacy

FDA Accepts Rocket Pharma’s BLA for LAD-I Treatment

The FDA accepted Rocket Pharmaceuticals’ (Cranbury, NJ) Biologics License Application (BLA) with Priority Review for RP-L201 (mametegragene autotemcel), a lentiviral vector-based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). LAD-I is a rare genetic immune disorder that increases the risk for patients to have recurrent and fatal infections. It is almost always fatal in childhood without an allogeneic hematopoietic stem cell transplant.

FDA Clears Cabaletta Bio’s IND for CABA-201 for Systemic Sclerosis

Cabaletta Bio (Philadelphia) reported its Investigational New Drug (IND) application for CABA-201 was given the go-ahead by the FDA for a Phase I/II trial in patients with systemic sclerosis. The drug is a 4-1BB-containing fully human CD19-CAR T cell therapy.

UroGen Plans Rolling NDA for UGN-102 to Begin January 2024

Princeton, NJ-based UroGen Pharma reached an agreement with the FDA on plans for submission of a New Drug Application (NDA) for UGN-102 (mitomycin) for an intravesical solution. The FDA agreed to a rolling review, allowing for early submission of the CMC section of the NDA, which is planned for January 2024.